FDA Approves Hepatic Veno-Occlusive Disease Treatment

The FDA approved defibrotide sodium (Defitelio) for the treatment of hepatic veno-occlusive disease in adults and children who have additional kidney or lung abnormalities following hematopoietic stem cell transplantation.

This is the first FDA-approved treatment for veno-occlusive disease.

“The approval of Defitelio fills a significant need in the transplantation community to treat this rare but frequently fatal complication in patients who receive chemotherapy and HSCT [hematopoietic stem cell transplantation],” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a FDA press release.

Defitelio was tested in 3 trials involving 528 patients who were diagnosed with hepatic veno-occulsive disease with liver or kidney abnormalities.

In the 3 studies, 38% to 45% of patients treated with Defitelio were alive 100 days after they received a stem cell transplant from blood or bone marrow.

The most common adverse effects associated with Defitelio include low blood pressure, nausea, diarrhea, vomiting, and nose bleeds. Other severe adverse effects have been observed in individuals, as well, including bleeding and allergic reactions.

The treatment should not be used in patients who have experienced bleeding complications or take blood thinners.